Clinical trial
Safety and Efficacy of Cyclophosphamide and Ruxolitinib for Graft-versus-host-disease Prophylaxis After Hematopoietic Stem Cell Transplantation With Thymoglobulin Serotherapy in Conditioning Regimen in Patients With Inborn Errors of Immunity
Name
IEI-PTCy 2023
Description
The aim of the current study is to evaluate the efficacy of combined regimen of GVHD prophylaxis with thymoglobulin in conditioning regimen and PTCY with ruxolitinib used after HSCT in patients with inborn errors of immunity (IEI)
Trial arms
Trial start
2023-11-21
Estimated PCD
2026-11-21
Trial end
2027-12-31
Status
Recruiting
Phase
Early phase I
Treatment
Cyclophosphamide
Cyclophosphamide 25mg/kg (days +3, +4) after HSCT from MUD and MRD Cyclophosphamide 50mg/kg (days +3, +4) after HSCT from MMRD
Arms:
intervention/treatment
Ruxolitinib
Ruxolitinib 7 mg/m2 from day +5 after HSCT
Arms:
intervention/treatment
Size
100
Primary endpoint
Event-free survival
1 year after HSCT
Eligibility criteria
Inclusion Criteria:
1. Patients aged ≥ 0 months and \< 21 years
2. Patients diagnosed with NBS eligible for an allogeneic HSCT
3. Signed written informed consent signed by a parent or legal guardian
Exclusion Criteria:
Concomitant severe somatic disease associated with an additional risk of severe complications
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 100, 'type': 'ESTIMATED'}}
Updated at
2024-01-10
1 organization
1 product
1 drug
1 indication
Drug
cyclophosphamideIndication
Primary ImmunodeficiencyProduct
Ruxolitinib